Marisa Wexler, MS, senior science writer —

Radicava (edaravone) significantly prolongs long-term survival for people with amyotrophic lateral sclerosis (ALS), according to an analysis of nearly 3,000 patients. It didn’t significantly slow the progression of disease symptoms, as assessed by scores on the ALS Functional Rating Scale-Revised (ALSFRS-R), however. The results were presented at last…

It’s been nearly three years since the HEALEY platform clinical trial started and, so far, two promising experimental treatments for amyotrophic lateral sclerosis (ALS) have been identified. Both potential medications — CNM-Au8 and pridopidine — are headed toward Phase 3 clinical testing, while testing has been ruled…

Treatment with the investigational therapy DNL343 was generally well-tolerated among people with amyotrophic lateral sclerosis (ALS) in an early clinical trial, and biomarker data from the trial suggest that the therapy is working as intended. Results were presented at the annual meeting of the American Academy of Neurology (AAN),…

Cold temperatures can activate a complex called the proteasome that cells use to clear themselves of unneeded proteins, including the protein clumps associated with amyotrophic lateral sclerosis (ALS), a new study reports. In cell models of the disease, exposure to cold temperatures for a prolonged time significantly lowered the…

AI Therapeutics‘ experimental therapy AIT-101 led to reductions in levels of toxic proteins in people with amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, according to data from a clinical trial. The Phase 2a clinical trial also met its main goals of showing that AIT-101…

An expanded access program for the experimental treatment SLS-005 has dosed its first participant, according to Seelos Therapeutics, the therapy’s developer. The expanded access program, or EAP, is an open-label study allowing access to SLS-005 for people with amyotrophic lateral sclerosis (ALS) who are…

TDP-43, a protein that is known to have toxic effects in amyotrophic lateral sclerosis (ALS), is able to interact with RNA molecules carrying a chemical modification called m6A, and this chemical modification is increased in ALS nerve cells, a new study shows. The findings add to a growing body…

Difficulties in measuring the progression of amyotrophic lateral sclerosis (ALS) among patients with advanced disease can cause complications in clinical trials, according to a new analysis that highlights how such problems may have affected the failed NurOwn study. The work was led by scientists at BrainStorm…

In most cases of amyotrophic lateral sclerosis (ALS), dysfunction of a protein known as TDP-43 leads to abnormally low levels of another protein, stathmin-2, which is sufficient to drive nerve cell dysfunction, new data suggest. In an aim to restore stathmin-2 levels, researchers created a designer DNA treatment. Now,…

On average, a person with amyotrophic lateral sclerosis (ALS) will require a wheelchair to get around within two years of the initial onset of disease symptoms. Findings underscore the condition’s rapid advance, and the need for new ALS treatments that work to slow disease progression, its researchers noted.